Janssen Seeks FDA Approval for Kinase Inhibitor to Treat Metastatic, Genetically-Altered Bladder Cancer


The kinase inhibitor is already approved for patients who experienced recurrence following 1 or more lines of standard of care platinum-containing chemotherapy.

Janssen Pharmaceutical Companies has submitted a supplemental New Drug Application (sNDA) to the FDA seeking approval for erdafitinib (Balversa; Janssen), a kinase inhibitor found to reduce the risk of death from metastatic urothelial carcinoma (mUC) by 36% compared to standard of care chemotherapy in the phase 3 THOR study.

Image credit: freshidea - stock.adobe.com

Image credit: freshidea - stock.adobe.com

The new drug is indicated for adult patients with locally advanced or mUC, who are at risk of fibroblast growth factor receptor (FGFR)3 genetic alterations. The indicated patient population also includes those who experienced disease progression during or following:

  • therapy with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the locally advanced or metastatic setting
  • 12 months of neoadjuvant or adjuvant therapy

“Erdafitinib continues to generate promising clinical findings for patients with FGFR-altered mUC, who often face poor disease outcomes," said Peter Lebowitz, MD, PhD, Global Therapeutic Area head, Oncology, Janssen Research & Development, LLC, in a press release.

The phase 3 randomized, open-label, and multicenter THOR (NCT03390504) study evaluated the efficacy and safety of erdafitinib against chemotherapy. The primary endpoint was overall survival (OS), and secondary endpoints were progression-free survival (PFS), objective response rate (ORR), duration of response (DOR), patient-reported outcomes, safety, and pharmacokinetics (PK).

Erdafitinib was significantly superior to chemotherapy at the interim analysis, reducing risk of death by 36% and being used as evidence to support the sNDA. The study was stopped based on these results and patients in the chemotherapy arm were able to receive erdafitinib. The safety profile was also consistent with previous data.

UC accounts for more than 90% of bladder cancers, and 20% of patients with UC have mUC or unresectable disease. Approximately 20% of patients with mUC have an FGFR alteration, which promotes tumor cell growth and survival, worsening survival outcomes. Those with late-stage metastatic disease have a 5-year survival rate of 8%.

The FDA granted erdafitinib Breakthrough Therapy Designation in 2018. In 2019, the FDA granted erdafitinib accelerated approval to treat adults with locally advanced or mUC who are susceptible to FGFR3 or FGFR2 genetic alterations and experienced disease progression during or following treatment with platinum-containing chemotherapy, including adjuvant or neoadjuvant.

The FDA needs to continue seeing clinical benefit in confirmatory trials to approve erdafitinib for this indication. New trials are evaluating erdafitinib for recurrent high-risk, non-muscle-invasive bladder cancer and non-muscle invasive or muscle invasive bladder cancer with FGFR alterations.

"Through the ongoing development of this targeted therapy, we are committed to transforming bladder cancer treatment to positively impact the lives of patients," Lebowitz said in the press release.


Janssen Submits Supplemental New Drug Application to the U.S. Food and Drug Administration Seeking Full Approval of BALVERSA® (erdafitinib) for the Treatment of Patients with Locally Advanced or Metastatic Urothelial Carcinoma and Selected Fibroblast Growth Factor Receptor Gene Alterations. Janssen. News Release. August 28, 2023. Accessed on August 29, 2023. www.janssen.com

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